RESEARCH TRIANGLE PARK – The potential for gene therapy to actually cure debilitating and deadly genetic disorders got another window opened today with a collaboration between two cutting-edge companies from North Carolina and the Boston area.

They see promise, without making promises. That’s because their science-based optimism will require years of research and clinical trials, and millions of dollars of investment without return. But it’s so compelling that they’re going to merge two titillating platform technologies they believe, when linked together, can skirt one of the biggest remaining problems that has hampered the rollout of gene therapy: the inability to add some kind of “do-over” or even increase a dose, if the first try wimps out.


North Carolina’s privately held Asklepios BioPharmaceutical, Inc. (AskBio), in Research Triangle Park, and publicly traded Selecta Biosciences, Inc. (NASDAQ: SELB), in Watertown, Mass., went public today with a strategic partnership to jointly develop, manufacture and commercialize therapeutics for what they believe might be life-changing, next-generation adeno-associated virus (AAV) gene therapies for people with few reasonable options for quality life, or survival.

The companies say the partnership will leverage both of their unique proprietary technology platforms with human proof-of-concept clinical trials to validate the potential for heretofore impossible “re-dosing” in gene therapy patients suffering from genetic diseases.

AskBio, perhaps the most important “unknown” corporation of the 21st century, as a North Carolina-based global pacesetter in unprecedented technology, is the brainchild of gene therapy pioneers representing backgrounds of compelling diversity: Richard Jude Samulski, Ph.D. the first scientist to clone AAV, president and chief scientific officer; Xiao Xiao, who was the first to develop a miniaturized dystrophin gene enabling the advancement of gene therapy for Duchenne Muscular Dystrophy; Josh Grieger, Ph.D., chief technology officer, who pioneered a production technology, the Pro10 cell line, that is paving the way to ensure these important gene therapeutics can reach all patients; and Sheila Mikhail, J.D. She’s a talented life science business leader who serves as board chair and chief executive officer.

AskBio’s pipeline. (AskBio image)

Samulski became a science legend when he discovered how AAV could be safely used to deliver corrected genes to cells with genetic defects. He was recruited to the University of North Carolina School of Medicine from the University of Pittsburgh in 1993 with the help of about $250,000 in grant funding from the North Carolina Biotechnology Center. He directed the Gene Therapy Center at UNC-Chapel Hill for years while he fine-tuned his lab, stacked up discoveries and locked in patents.

Through it all, grants and loans from the Biotech Center supported the development of Samulski’s academic research and AskBio’s commercial technologies respectively. NCBiotech specialists recognized early that gene therapy was poised to become a human health game-changer.

They were right. AAV has emerged as an efficient, safe, and versatile vehicle for delivering corrective genes inside of the body. Significantly, the AskBio platform was used to develop the only two FDA-approved gene therapies available today: Zolgensma and Luxterna.

AskBio, created as one of the first gene therapy companies in the world, now owns more than 500 patents and applications for AAV technology and processes. AskBio’s gene therapy platform includes not only a robust pipeline of potentially curative gene therapies. The company has also established a groundbreaking manufacturing process that attracts suitors and partners, like Selecta, from around the world.

AskBio gets $235 million in gene therapy support

AskBio also has several advanced AAV initiatives under development, including one named “Doggybone DNA.”

“We only seek to collaborate with companies that share our mission and core values, which are focused on one goal, finding the answers that will cure patients suffering from life-threatening genetic diseases,” said Mikhail. “Selecta is a leading example of that kind of company, and we’re enthusiastic to have this opportunity to work with them on behalf of patients and their families.”

Samulski added, “We look forward to this opportunity to deploy our collective platforms to overcome one of the key obstacles to providing long-term treatment from AAV-based therapeutics to all patients.”

Selecta plans to combine its immune tolerance technology (ImmTOR) platform with a range of biologic therapies for rare and serious diseases that require new treatment options because they provoke unwanted agitation of the body’s immune system – a process called immunogenicity.

The company’s pipeline includes several ImmTOR-powered therapeutic enzyme and gene therapy product candidates. It’s developing its lead candidate, SEL-212, to treat gout. Its gene therapy candidates, in preclinical development for certain rare inborn errors of metabolism, incorporate ImmTOR with the goal of addressing barriers to repeat administration.

(C) N.C. Biotech Center